BPC January 24 update

15 key near-term biotech stock catalysts - likely 1Q 2019

Weekly watchlist

 At the beginning of the year we noted a number key data readouts due this year. With the first month of 1Q almost at an end, we highlight key readouts slated by the end of March, including a couple for 1H 2019 that might readout this quarter. It is by no means a full list but these events will be closely watched. Included in the list are four key PDUFA dates from the list noted last week.

First, let’s review the week that was in what was a relatively quiet shortened trading week.

Editas Medicine, Inc. (NASDAQ: EDIT) shares closed Tuesday down 20% to $20.95 following news its CEO, Katrine Bosley, resigned as President and CEO. Shares of other CRISPR companies also fell on the news with CRISPR Therapeutics AG (NASDAQ: CRSP) closing down 11% to $33.08, while shares of Intellia Therapeutics, Inc. (NASDAQ: NTLA) closed down 7% to $14.16. 

Bristol-Myers Squibb Company (NYSE:BMY) announced Thursday that following discussions with the FDA it has withdrawn its supplemental Biologics License Application (sBLA) for the Opdivo and low-dose Yervoy for treatment of first-line advanced non-small cell lung cancer (NSCLC). Further data due 1H 2019 from its Phase 3 trial, Checkmate-227, will be required for analysis before resubmitting the application. 

AbbVie (NYSE:ABBV) shares closed Friday down 6% to $80.54 following the release of 4Q 2018 financial results. Investors sold down shares of the company on news that its main drug, Humira, saw its international sales decline by 14.8% due to competition from biosimilars.

Advaxis, Inc. (NASDAQ:ADXS) announced Wednesday the FDA has placed its Phase 3 trial of axalimogene filolisbac (AXAL) for the treatment of cervical cancer (AIM2CERV) on partial clinical hold. The hold follows requests for additional information pertaining to certain AXAL chemistry, manufacturing and controls (CMC) matters. All currently enrolled patients will continue to receive treatment, per the trial protocol. However, no new patients can enrol in AIM2CERV until resolution of this partial hold. 

A number of biopharma companies announced public offerings of common stock this latest week, while a couple also noted reverse stock splits.

Offerings: BLPH CRBP PTCT RNN SELB URGN WVE NCNA (later withdrawn)

Reverse stock splits: OHRP TTNP


Biotech stock near-term catalysts (likely 1Q 2019):

Drug Stage Catalyst Market Cap

ALNY – Alnylam Pharmaceuticals Inc.
Givosiran
Acute hepatic porphyrias

Phase 3 Phase 3 positive interim analysis reported September 27, 2018. Data due March 2019. Rolling NDA has been initiated.
$8.3 billion

GILD – Gilead Sciences Inc.
Selonsertib (GS-4997) - STELLAR 4
Nonalcoholic steatohepatitis (NASH)

Phase 3 Phase 3 trial did not meet primary endpoint - February 11, 2019.
$84.3 billion

ICPT – Intercept Pharmaceuticals Inc.
Ocaliva (Obeticholic acid (OCA)) - REGENERATE
Adult nonalcoholic steatohepatitis (NASH) patients.

Phase 3 Phase 3 data met one of two co-primary endpoints - February 19, 2019. Full data due at EASL April 10-14, 2019.
$2.8 billion

IMGN – ImmunoGen Inc.
Mirvetuximab soravtansine - FORWARD I
Cancer - ovarian cancer and relapsed endometrial cancer

Phase 3 Phase 3 top-line data due 1H 2019.
$854.3 million

JNJ – Johnson & Johnson
Esketamine
Treatment resistant depression

PDUFA priority review PDUFA estimate March 3, 2019 assuming priority review. FDA Advisory Committee Meeting February 12, 2019 voted 14-2 that benefits outweigh risks.
$366.4 billion

KPTI – Karyopharm Therapeutics Inc.
Selinexor
Quadruple Refractory Multiple Myeloma

PDUFA priority review PDUFA date under priority review April 6, 2019. Advisory Committee Meeting February 26, 2019 - 12:30pm. Briefing notes released February 22, 2019.
$307.8 million

LXRX – Lexicon Pharmaceuticals Inc.
Sotagliflozin
Type 1 Diabetes

PDUFA PDUFA date March 22, 2019. Advisory Committee Meeting January 17, 2019 voted 8-8 that benefits outweigh risks.
$583.6 million

MRK – Merck & Company Inc. (new)
KEYTRUDA and Inlyta - KEYNOTE-426
Renal cell carcinoma

PDUFA priority review PDUFA date under priority review June 20, 2019.
$210 billion

NVAX – Novavax Inc.
RSV vaccine - prepare trial
Respiratory Syncytial Virus (RSV) in healthy pregnant women - protect infants via maternal immunization

Phase 3 Phase 3 top-line data due 1Q 2019.
$735.1 million

PFE – Pfizer Inc.
PF-06939926
Duchenne muscular dystrophy (DMD)

Phase 1b Phase 1b initiation announced April 12, 2018. Early data due 1H 2019.
$248.3 billion

PRTO – Proteon Therapeutics Inc.
Vonapanitase (PRT-201) PATENCY-2
Chronic kidney disease (CKD) patients undergoing surgical placement of an arteriovenous fistula (AVF)

Phase 3 Phase 3 data due March 2019.
$57.7 million

PTI – Proteostasis Therapeutics Inc.
PTI-428 + PTI-801 + PTI-808
Cystic fibrosis

Phase 1 Phase 1 full data due 1Q 2019.
$205.3 million

REPH – Recro Pharma Inc.
Intravenous (IV) meloxicam
Acute pain following bunionectomy surgery

PDUFA CRL announced May 24, 2018. New PDUFA date March 24, 2019.
$179.6 million

SGEN – Seattle Genetics Inc.
Enfortumab vedotin - EV201
Urothelial cancer

Phase 2 Phase 2 pivotal data due 1Q 2019.
$11.4 billion

SRPT – Sarepta Therapeutics Inc.
SRP-5051 - PPMO
Duchenne muscular dystrophy

Phase 1/2 Phase 1 trial safety and dosing insight due 1Q 2019.
$9.4 billion